中文 |
English |
Email Alert
基于环糊精构建的基因载体进展
Recent Progress in Gene Delivery Based on Cyclodextrin
安全有效的基因载体对于基因治疗有着重要的潜在价值。相对于病毒性基因载体，化学合成的载体具有低免疫原性，易于大规模生产和生产成本较低的特性，因而受到越来越多的关注，但是非病毒基因载体在转染效率和选择性方面有一定的限制性，当前的主要研究工作集中在这两方面。基于环糊精构建的基因载体，可以有效地提供基因载体的立体构象和功能性的选择性。作为FDA批准的生物材料，环糊精具有无毒和生物降解性，其不但可以保护基因，避免在体内降解，同时有助于通过细胞膜，进入细胞内达到基因转染的作用。环糊精具有大量可修饰的羟基基团，因此对环糊精修饰不但可以通过主客体作用构建超分子体系，并且可以作为多官能团核形成星状高分子，被广泛应用于制备低毒、可降解、靶向性和高效率的转基因载体。目前，环糊精修饰的非病毒正离子载体转移siRNA，已经成功进行了色素瘤的临床试验，取得了很好的治疗效果，表明了环糊精非病毒载体的巨大应用前景。本文对基于环糊精基因载体的最新研究进展进行了综述，详细介绍了基于环糊精的超分子自组装构建的聚轮烷型、侧链分子识别型的基因载体，以及基于环糊精多羟基构型而构建的星型聚合物基因载体和树枝状基因载体，并对环糊精基因载体的优越性和未来应用做了相应的介绍。
Safe and efficient delivery of nucleic acid constructs to target cells has great potential for the treatment of genetic diseases. Non-viral gene delivery has attracted considerable attention because of large-scale production, immunogenicity and safety concerns. Whereas, non-viral gene delivery still suffered from low transfection efficiency and lack of selectivity, which is the main target on this field. The gene delivery based on cyclodextrin (CD) can offer the possibility for construction of various multi-functional gene deliveries. As a FDA approved bio-material, CDs have been extensively used for gene delivery because of their ability to stabilize the nucleic acids in biological media and their ability to destabilize and permeate biological membranes and for obviating undesirable side effects. CD is not only a widely used host molecule capable of internalizing guest molecules in water, but also a core with abundant OH groups. Therefore, a variety of nonviral vectors have been explored based on CD's host-guest interaction and core structure. Recently, CD modified gene delivery has achieved to the clinic test with remarkable result, showing great potential application. In this review the newest development of the gene delivery based on cyclodextrin is summarized. The article provides detail information about the rotaxanes type, side-chain type, star-shape type and branched type deliveries of nucleic acid. Furthermore, the advantage of CD based gene delivery is emphasized herein, which has been applied in clinical test.
Contents
1 Introduction
2 Gene delivery based on cyclodextrin
2.1 Rotaxane type gene deliveries
2.2 Side-chain type gene deliveries
2.3 Star-shape type gene deliveries
2.4 Branched type gene deliveries
2.5 Other type gene deliveries
3 Conclusion and outlook
cyclodextrin
[1] (a) Xu P, Kirk E A, Zhan Y, Murdoch W J, Radosz M, Shen Y. Angew. Chem. Int. Ed., 2007, 46: 4999; (b) Meager A. Gene "Therapy Technologies: Applications and Regulations", John Wiley & Sons Ltd. U. K. 1999.
[2] Mintzer M A, Simanek E E. Chem. Rev., 2009, 109: 259.
[3] Boussif O, Lezoualc H F, Zanta M A, Mergny M D, Scherman D, Demeneix B, Behr J P. Proc. Natl. Acad. Sci. U. S. A., 1995, 92: 7297.
[4] (a) Rekharsky M V, Inoue Y. Chem. Rev., 1998, 1875; (b) Liu L, Guo Q X. J. Inclu. Phenom. Macro. Chem., 2004, 50: 95; (c) Liu L, Guo Q X. J. Inclu. Phenom. Macro. Chem., 2002, 42: 1; (d) Liu L, Guo Q X. J. Phys. Chem. B, 1999, 103: 3461; (e) Mellet C O, Fernandez J M G, Benito J M. Chem. Soc. Rev., 2011, 40: 1586.
[5] Neu M, Fischer D, Kissel T. J. Gene. Med., 2005, 7: 992.
[6] (a) 董海青(Dong H Q), 李永勇(Li Y Y), 李兰(Li L), 时东陆(Shi D L). 化学进展(Prog. Chem.), 2011, 23: 914; (b) 周冬香(Zhou D X), 孙涛(Sun T), 邓维(Deng W). 有机化学(Chin. J. Org. Chem.), 2012, 32: 239.
[7] (a) Lee S C, Choi H S, Ooya T, Yui N. Macromolecules, 2004, 37: 7464.; (b) Choi H S, Yamamoto K, Ooya T, Yui N. Chem. Phys. Chem., 2005, 6: 1081.; (c) Choi H S, Hirasawa A, Ooya T, Kajihara D, Hohsaka T, Yui N. Chem. Phys. Chem., 2006, 7: 1671; (d) Joung Y K, Choi H S, Ooya T, Yui N. J. Inclusion Phenom. Macrocyclic Chem., 2007, 57: 323.
[8] Shuai X T, Merdan T, Unger F, Kissel T. Bioconjug. Chem., 2005, 16: 322.
[9] (a) Li J, Yang C, Li H, Wang X, Goh S, Ding J, Wang D, Leong K. Adv. Mater., 2006, 18: 2969; (b) Yang C, Wang X, Li H, Goh S H, Li J. Biomacromolecules, 2007, 8: 3365; (c) Li J, Loh X J. Adv. Drug Deliv. Rev., 2012, 60: 1000; (d) Li Z, Yin H, Zhang Z, Liu K L, Li J. Biomacromol., 2012, 13: 3162; (e) Yang C, Wang X, Li H, Tan E, Lim C T, Li J. J. Phys. Chem. B, 2009, 113: 7903.
[10] (a) 李春鸽(Li C G), 赵爽(Zhao S), 李俊杰(Li J J), 尹玉姬(Yin Y J). 化学进展(Prog. Chem.), 2013, 25: 122; (b) 尤树森(You S S), 杨万泰(Yang W T), 尹梅贞(Yin M Z). 化学进展(Prog. Chem.), 2012, 24: 2198; (c) 沈银(Shen Y), 胡桂香(Hu G X), 张华星(Zhang H X), 齐莉莉(Qi L L), 骆成才(Luo C C). 化学学报(Acta Chimica Sinica), 2013, 71: 323.
[11] Yamashita A, Kanda D, Katoono R, Yui N, Ooya T, Maruyama A, Akita H, Kogure K, Harashima H. J. Control. Release, 2008, 131: 137.
[12] Ralfkirche I S, Lionel W, Wanger E. Adv. Drug. Deliver. Rev., 2001, 53: 341.
[13] Zhou Y, Wang H, Wang C, Li Y, Lu W, Chen S, Luo J, Jiang Y, Chen J. Mol. Pharmaceutics, 2012, 9: 1067.
[14] Kulkarni A, DeFrees K, Schuldt R A, Vlahu A, VerHeul R, Hyun S, Deng W, Thompson D H. Integr. Biol., 2013, 5: 115.
[15] Liu Y, Yu L, Chen Y, Zhao Y L, Yang H. J. Am. Chem. Soc., 2007, 129: 10656.
[16] Burckbuchler V, Wintgens V, Leborgne C, Lecomte S, Leygue N, Scherman D, Kichler A, Amiel C. Bioconjugate Chem., 2008, 19: 2311.
[17] Zhang J, Sun H, Ma P X. ACS Nano, 2010, 4: 1049.
[18] Liu Y, Yu Z, Zhang Y, Guo D, Liu Y. J. Am. Chem. Soc., 2008, 130: 10431.
[19] Buckwalter D J, Sizovs A, Ingle N P, Reineke T M. ACS Macro Lett., 2012, 1: 609.
[20] Deng W, Chen J, Kulkarnia A, Thompson D H. Soft Matter, 2012, 8: 5843.
[21] Kulkarni A, Deng W, Hyun S, Thompson D H. Bioconjugate Chem., 2012, 23: 933.
[22] (a)Aloorkar N H, Kulkarni A S, Patil R A, Ingale D J. Int. J. Pharm. Sci. Nanotechnol., 2012, 5: 1675; (b) 付云(Fu Y), 王海蛟(Wang H J), 张骥(Zhang J), 余孝其(Yu X Q). 化学学报(Acta Chimica Sinica), 2013, 71(04): 585.
[23] Huang H, Tang G, Wang Q, Li D, Shen F, Zhou J, Yu H. Chem. Commun., 2006, 2382.
[24] (a) Srinivasachari S, Fichter K M, Reineke T M. J. Am. Chem. Soc., 2008, 130: 4618; (b) Sizovs A, McLendon P M, Srinivasachari S, Reineke T M. Top. Cur. Chem., 2010, 131; (c) Srinivasachari S, Reineke T M. Biomaterials, 2009, 30: 928.
[25] (a) Bennevault-Celton V, Urbach A, Martin O, Pichon C, Guégan P, Midoux P. Bioconjugate Chem., 2011, 22: 2404; (b) Bertrand E, Goncalves C, Billiet L, Gomez J P, Pichon C, Cheradame H, Midoux P, Guegan P. Chem. Commun., 2011, 47: 12547.
[26] (a) Zhao F, Yin H, Zhang Z, Li J. Biomacromolecules, 2013, 14: 476; (b) Wang X, Li J, Wang Y, Koenig L, Gjyrezi A, Giannakakou P, Shin E H, Tighiouart M, Chen Z, Nie S. ACS Nano, 2011, 5: 6184; (c) Wang X, Li J, Wang Y, Cho K J, Kim G, Gjyrezi A, Koenig L, Giannakakou P, Shin H J C, Tighiouart M, Nie S, Chen Z, Shin D M. ACS Nano, 2009, 3: 3165; (d)Fang G M, Li Y M, Shen F, Huang Y C, Li J B, Lin Y, Cui H K, Liu L. Angew. Chem. Int. Ed., 2011, 50: 7645.
[27] (a) Xu F J, Zhang Z X, Ping Y, Li J, Kang E T, Neoh K G. Biomacromolecules, 2009, 10: 285; (b) Liu M, Li Z H, Xu F J, Lai L H, Wang Q Q, Tang G P, Yang W T. Biomaterials, 2012, 33: 2240.
[28] (a) Hu Y, Zhu Y, Yang W T, Xu F J. ACS Appl. Mater. Interfaces, 2013, 5: 703; (b) Xiu K M, Yang J J, Zhao N N, Li J S, Xu F J. Acta Biomater., 2013, 9: 4726; (c) Hu Y, Zhu Y, Yang W T, Xu F J. ACS Appl. Mater. Interf., 2013, 5: 703.
[29] (a) Meéndez-Ardoy A, Guilloteau N, Giorgio C D, Vierling P, Santoyo-Gonzlez F, Mellet C O, Fernndez J M G. J. Org. Chem., 2011, 76: 5882; (b) Bienvenu C, Martinez A, Jimenez Blanco J L, Di Giorgio C, Vierling P, Ortiz Mellet C, Defaye J, Garcia Fernandez J M. Organ. Biomol. Chem., 2012, 10: 5570; (c) Diaz-Moscoso A, Guilloteau N, Bienvenu C, Mendez-Ardoy A, Jimenez Blanco J L, Benito J M, Le Gourrierec L, Di Giorgio C, Vierling P, Defaye J. Biomaterials, 2011, 32: 7263; (d) Diaz-Moscoso A, Le Gourrierec L, Gomez-Garcia M, Benito J M, Balbuena P, Ortega-Caballero F, Guilloteau N, Di Giorgio C, Vierling P, Defaye J. Chem. Euro. J., 2009, 15: 12871.
[30] (a) Villari V, Mazzaglia A, Darcy R, O'Driscoll C M, Micali N. Biomacromolecules, 2013, 14: 811; (b) O'Mahony A M, Godinho B M D C, Ogier J, Devocelle M, Darcy R, Cryan J F, O'Driscoll C M. ACS Chem. Neurosci., 2012, 3: 744.
[31] (a) 何谷 (He G), 郭丽(Guo L). 有机化学(Chin. J. Org. Chem.), 2008, 8: 1326; (b) 董博(Dong B), 闫熙博(Yan X B), 牛玉洁(Niu Y J), 王欣(Wang X), 王连永(Wang L Y), 王燕铭(Wang Y M). 化学进展(Prog. Chem.), 2012, 24: 2352.
[32] (a) Kihara F, Arima H, Tsutsumi T, Hirayama F, Uekama K, Bioconjugate Chem., 2002, 13: 1211; (b) Kihara F, Arima H, Tsutsumi T, Hirayama F, Uekama K. Bioconjugate Chem., 2003, 14: 342; (c) Arima H, Arizono M, Higashi T, Yoshimatsu A, Ikeda H, Motoyama K, Hattori K, Takeuchi T, Hirayama F, Uekama K. Cancer Gene Therapy, 2012, 19: 358; (d) Ikeda Y, Motoune S, Ono M, Arima H, Hirayama F, Uekama K. J. Drug Deliv. Sci. Tech., 2004, 14: 69.
[33] (a) Chen Y, Zhou L Z, Pang Y, Huang W, Qiu F, Jiang X L, Zhu X Y, Yan D Y, Chen Q. Bioconjugate Chem., 2011, 22: 1162; (b) Liu Y, Yu C, Jin H, Jiang B, Zhu X, Zhou Y, Lu Z, Yan D. J. Am. Chem. Soc., 2013, 135: 4765; (c) Dong R, Chen H, Wang D, Zhuang Y, Zhu L, Su Y, Yan D, Zhu X. ACS Macro Letters, 2012, 1: 1208; (d) Dong R, Liu Y, Zhou Y, Yan D, Zhu X. Polymer Chem., 2011, 2: 2771; (e) Dong R, Zhou L, Wu J, Tu C, Su Y, Zhu B, Gu H, Yan D, Zhu X. Chem. Commun., 2011, 47: 5473.
[34] (a) Park I K, von Recum H A, Jiang S, Pun S H. Langmuir, 2006, 22: 8478; (b) Pun S H, Bellocq N C, Liu A, Jensen G, Machemer T, Quijano E, Schluep T, Wen S, Engler H, Heidel J, Davis M E. Bioconjugate Chem., 2004, 15: 831; (c) Bellocq N C, Kang D W, Wang X, Jensen G S, Pun S H, Schluep T, Zepeda M, Davis M E. Bioconjugate Chem., 2004, 15: 1201.
[35] (a) Davis M E, Zuckerman J, Choi C H, Seligson D, Tolcher A, Alabi C, Yen Y, Heidel J, Ribas A. Nature, 2010, 464: 1067; (b) Hwang S J, Bellocq N C, Davis M E. Bioconjugate Chem., 2001, 12: 280; (c) Pun S H, Davis M E. Bioconjugate Chem., 2002, 13: 630; (d) Pun S H, Bellocq N C, Liu A, Jensen G, Machemer T, Quijano E, Schluep T, Wen S, Engler H, Heidel J, Davis M E. Bioconjugate Chem., 2004, 15: 831; (e)Kulkarni R P, Mishra S, Fraser S E, Davis M E. Bioconjugate Chem., 2005, 16: 986.
/
| 〈 |
|
〉 |
PDF
(3820KB)
